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Jun 1, 2015

As FDA notes at the outset of the new draft guidance document, "Sponsor-investigators seeking to do clinical research often do not have the regulatory knowledge or the resources to hire experts to help them with the IND submission process."

The guidance document is therefore intended to act as a basic overview of the IND process with an eye on individual researchers investigating new uses for either an approved or investigational drug.

FDA's guidance goes on to explain the basics of 21 CFR 312—the federal regulation governing IND submissions.

For example, FDA provides an overview of the essential elements of an IND submission, including information about the sponsor, a summary of the investigational drug product and its risks, the clinical trial protocol and a summary of previous clinical trial results involving the drug.

The guidance, FDA notes, is not intended for use by sponsors or investigators "seeking to evaluate a drug for commercial purposes (i.e. seeking market approval or licensure) and thus does not focus on certain regulatory requirements that involve exchange of information or materials between a sponsor and investigator."

The inspectional data cover all aspects of FDA’s BIMO program (i.e., clinical investigators, IRBs, sponsors, bioequivalence, and good laboratory practices) for all Centers, as applicable. These data were provided by the individual Centers and may differ from other inspection data available on FDA’s website, as different criteria and/or methods for compiling the information may have been used. Also provided, at the first link, is a glossary of acronyms.

Beat Widler, Managing Partner, Widler & Schiemann AG, Zug, Switzerland highlighted in his presentation reasons why clinical studies go wrong and measures to get them right.

The FDA, which has successfully reduced the review times for new medications, wants to help biopharmaceutical companies lower the rising costs of clinical trials by streamlining some of its processes and working more closely with industry. That’s the proposal Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research (CDER), outlined to senators on the Health Education Labor and Pensions committee end of April 2015.

In Europe, gene therapies are regulated as advanced therapy medicinal products (ATMPs) under Regulation (EC) No. 1394/2007, and are further defined in Part IV of Annex I to Directive 2001/83/EC. Thus far, EMA has only authorized a single gene therapy, Glybera, which treats a rare condition called lipoprotein lipase deficiency (LPLD).

The EMA aims to support and facilitate the development of these innovative medicines by giving guidance to developers on the types of evidence they should generate to support a marketing-authorization application with a regulatory authority in the European Union.